Q1-Gene therapy research aims to use genetic material (DNA or RNA) to develop
effective new treatments for a range of serious genetic conditions.
Critically discuss the strategy you would use to develop a gene therapy
treatment for a named monogenic disorder. Your answer should include
details of the therapeutic genetic material, how you would deliver this to
the affected cells, and the reasons for your choice.
Evaluate the regulatory challenges and potential safety issues that would
need to be overcome before your new gene therapy treatment could be
used to treat patients.
a.) Early gene therapy failed to achieve long-term expression of genes implemented with the aim of removing or disrupting defective genes due to monogenetic disorders. Currently, Viruses are used in gene therapy because they can transfer genes into human cells. When a virus becomes a "vector," it is capable of transmitting the desired gene into cells but not of taking over or damaging the cells. Retroviruses, adenoviruses, and adeno-associated viruses are among the viruses commonly used in gene therapy.
If a defective gene causes a required protein to be faulty or absent, gene therapy may be able to restore the protein's function by introducing a regular copy of the gene. In most cases, a gene inserted directly into a cell will not survive. Instead, the gene is delivered by a genetically modified carrier called a vector. Since they can deliver the new gene by infecting the cell. When the viruses are used in humans, they are changed so that they do not cause disease. Retroviruses, for example, insert their genetic material (which includes the new gene) into a chromosome in the human cell.
b.) Adeno-associated virus (AAV) vectors are one of the most widely used viral vectors for gene therapy today. AAV is a non-enveloped virus with a single-stranded single-stranded DNA genome. AAVs have a number of advantages. They only cause minor immune reactions, and different AAV serotypes can be used to deliver genes to a variety of tissues. They infect both dividing and non-dividing cells, but do not incorporate genetic material into the host genome. The genetic material delivered by AAV normally stays outside the genome, acting as a guide for protein transcription in the cell.
AAV's potential as a delivery vehicle for gene therapy applications has grown due to the virus's lack of pathogenicity, longevity, and wide range of serotypes. The vector can be injected or administered intravenously (IV) into a particular body tissue, where it is taken up by individual cells. In a laboratory environment, a sample of the patient's cells can be extracted and exposed to the vector. After that, the vector-containing cells are returned to the patient. If the treatment is successful, the vector's new gene will produce a functional protein.
Until gene therapy may be used to cure disease, researchers must clear a number of technological hurdles. For example, scientists must devise new methods for delivering genes and directing them to particular cells. They must also ensure that new genes are precisely monitored by the body in order to prevent further damage or undesirable mutations.